Researchers have developed a new minipig model of juvenile Batten disease that recapitulates some key features of the human condition, including vision loss and gait abnormalities. Despite certain limitations, scientists believe these miniswine are more translatable to the human disease compared with mouse models of juvenile Batten, also known…

A mini pig model could be an effective way to examine brain changes to track disease progression in late infantile Batten disease, a study suggests. In addition, measuring changes in the size of specific brain regions may help identify response to potential treatments, researchers note. “Miniswine present a…

A single intravitreal injection — into the vitreous cavity, in the back of the eye — of a gene therapy was found to preserve the function and structure of the retina in a dog model of late infantile Batten disease. Eye inflammation was…

CLN7, the protein lacking in CLN7 Batten disease, is involved not only in the functioning of lysosomes (the cellular compartment responsible for breaking down waste) but also in the communication between nerve and muscle cells, according to a preclinical study. These findings open the possibility that proteins associated with other…

Researchers have found a natural model of CLN7 disease, a rare form of Batten disease, in a type of monkey called a macaque, which may prove valuable in gaining insight into the disease, as well as testing therapeutic strategies, a study reports. The study, “Discovery…